Companies related to rare diseases

Driving change to defeat serious chronic diseases, built upon our heritage in diabetes.

The essential biodata life science partner for rare and neurodegenerative diseases

AOP Health is the European pioneer for integrated therapies for rare diseases and in critical care.

Our vision is to harness the power of the immune system to develop novel therapies against cancer & infectious diseases.

If you have kids, be glad you have Children's.

We’re working to enable faster and deeper genomic diagnosis & research, to bring genomic healthcare to all who need it.

For Healthier GENerations

Pharmaceutical Company & Service Partner

Our mission is to build a healthier tomorrow for patients with rare and serious liver diseases.

Decoding Secreted Proteins

Commercialization of innovative therapies in Central and Eastern Europe

Alone we are rare. Together we are strong.®

We Deliver on Your Promises

Navigating the Middle East & Africa to Broaden Patient Access, Faster

Better Bioinformatics For Everyone!

Specialty Plasma-Derived Therapeutics

Always Ahead

Helping bring new therapies to light

Variantyx is a technology-driven precision medicine company providing state-of-the-art genomic testing.

A clinical-stage international biotechnology research company focused on treatment innovation for mitochondrial disease

Developing innovative therapeutics for diseases at confluence of fibrosis and inflammation with high unmet need

Scientific support for healthcare

Powering the Future of Genetic Medicines

BCDD is a fully integrated center for translational research providing support for early-stage drug discovery.

Working across borders and diseases to improve the lives of all people living with rare diseases.

Генетичната лаборатория осъществява високоспециализирана болнична и доболнична генетична помощ.

Unlocking access to rare #AccessRare

Transforming Lives, Every Day.

Imagine faster patient access to medicines and improved health outcomes with Clinerion real world data.

Making it accessible professional and quality service for pharmaceutical import and global outsourcing.

Clinical-stage biopharma company developing novel RNA-modulating drug candidates to treat rare and ultra rare diseases.

Open source and open access innovation in healthcare knowledge - a new publishing paradigm.

Helping seriously ill children lead a more marvellous life.

Healthcare marketing specialists in CEE region.

Transforming Healthcare: Leading the Way in Pharmaceutical Sales, Marketing, Storage, and Distribution in Iraq.

INTERGEN Official Account (INTERGEN Resmi Hesabı)

Institut Imagine, pour guérir les maladies génétiques.

We develop oral small-molecule and protein therapeutics to target complement-mediated and other rare diseases.

We are the forefront of medical research, solving the greatest health problems facing our community.

Decoding Biology to Radically Improve Lives

Your trusted business partner for un-licensed & licensed medicines market in Turkey!

Face2Gene - Detecting Rare Disease Early with the Help of AI

Providing relief to patients with unmet medical needs in select specialty and rare diseases.

AI-Powered Clinical Research | Strategic & Synergetic Scaling™ | Data-Driven CRO Excellence

Working together to find a cure

Tenacious in our search for transformative therapies

India's Pioneering Genomics Company: Preventive Genomics (Genomepatri, MyFitGene, MedicaMap) + Clinical (WES, WGS etc)

Great Science Deserves Great People.

AI for Faster Rare Disease Diagnosis | Scalable PaaS Solutions for Hospitals & Pharma

AI for Faster Rare Disease Diagnosis | Scalable PaaS Solutions for Hospitals & Pharma

AI for Faster Rare Disease Diagnosis | Scalable PaaS Solutions for Hospitals & Pharma

AI for Faster Rare Disease Diagnosis | Scalable PaaS Solutions for Hospitals & Pharma

Developing Novel, First in Class Drug, Targeting Type 2 Diabetes, Based on a Core-Technology-Platform

Transforming life sciences with cutting-edge proteomics technology and solutions

Pharmaceutical and Healthcare Market Research Consultants

We develop breakthrough treatments for Claudin1-positive tumors and organ fibrosis

Developing best-in-class curative gene therapies for rare disorders.

Improve diagnosis, treatment and care of people affected by rare neurological diseases in Europe.

Advancing our proprietary Axiomer™ RNA-editing platform technology

Developing and commercializing novel therapies for rare genetic skin diseases with no approved treatments.

Capricor (NASDAQ: CAPR) is a biotechnology company developing cell and exosome-based therapeutics for serious diseases.

Sleep and wakefulness, both of them, when immoderate constitute disease

Smart Clinical Operations. Tailored partnerships. Real results.

Independent pharmacy offering specialized services, medications and treatments

Approaching healthcare innovation with purpose and agility to deliver new breakthroughs for people facing disease.

Improving the accessibility to the ERNs by integrating them into the National Healthcare Systems

Your Orphan Drug Companion!

Lipotype is the leading lipidomics service provider.

European Reference Network (ERN) for rare urogenital diseases & complex conditions requiring highly-specialised surgery

What If You Could Repair Broken Genes?

Empresa biotecnológica en Sevilla basada en el desarrollo de herramientas de ayuda al diagnóstico.

Advancing a targeted approach to treat GRIN-related disorders and other pediatric neurodevelopmental disorders.

A BETTER CHANCE AT LIFE

Our Vision : We have High Standards - We Build Companies - We have Impact

Developing transformative gene therapies for patients suffering from severe rare diseases.

Dedicated to transforming the lives of patients with severe and other genetic diseases through innovative gene therapy.

Supply chain specialist for rare & orphan diseases' medicines in the pharmaceutical industry

Until the cure of the rare disease of Pulmonary Hypertension we will be here to help any patient around the world

Developing drugs for orphan CNS diseases

Accessible Innovations

Pioneering a powerful new class of medicines based on red blood cell extracellular vesicles

No rare journey alone. Previously known as Findacure.

Maximizing Your Brand’s Potential Through Data-Driven Solutions

At Mendelian, rare diseases are our priority. We're enabling doctors to help patients earlier by finding the undiagnosed

Institut Imagine, pour guérir les maladies génétiques.

We're on a mission to help diagnosis and get new drug treatments into the hands of rare disease patients. Quickly.

Assessment and #Neurorehabilitation Platform for professionals featuring thousands of #CognitiveStimulation activitie

Making promising innovations possible together

We are on a global mission to establish gene therapy as a new standard of care for the leading causes of vision loss.

Helping Bring Science to Life

We create value in the field of medical diagnostics & biotechnology and move forward with innovative products.

A novel technology platform for the delivery of therapeutics across biological barriers

AI-Driven Diagnosis of Rare Diseases.

Where creative thinking meets scientific excellence

An initiative for the integration of genetic and genomic data in medicine and science throughout Austria.